Pyrukynd
Pyrukynd (mitapivat) is a pyruvate kinase activator. It's FDA-approved to treat hemolytic anemia in adults with pyruvate kinase deficiency. Pyrukynd (mitapivat) is taken by mouth twice daily with or without food. Some common side effects of this medication include back and joint pain.
What is Pyrukynd (mitapivat)?
What is Pyrukynd (mitapivat) used for?
Hemolytic anemia in adults with pyruvate kinase (PK) deficiency
How Pyrukynd (mitapivat) works
Pyruvate kinase (PK) is an enzyme (protein) that gives your red blood cells (RBCs) energy to travel throughout your body. If you have PK deficiency, your PK enzyme doesn't work well. This means your RBCs break down more quickly than they should, which results in lower RBC levels (anemia).
Pyrukynd (mitapivat) is a pyruvate kinase (PK) activator. It works by turning on PK enzymes. This means your RBCs won't break down as quickly and will have the energy needed to bring oxygen throughout the body.
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What are the side effects of Pyrukynd (mitapivat)?
Common Side Effects
- Back pain (15%)
- Joint pain (10%)
- High triglyceride (fat) levels (8%)
- Nausea, vomiting, or other symptoms of viral stomach infection (8%)
- Pain in mouth and throat (8%)
- Hot flashes (8%)
Other Side Effects
- Irregular heart rhythm
- Higher blood pressure
- Breast discomfort
- Dry mouth
- "Pins-and-needles" feeling
Serious Side Effects
- Short-term hemolytic anemia: yellowing of skin or eyes, dark urine, dizziness, confusion, fatigue, shortness of breath
Source: DailyMed
The following side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Hemolytic anemia—unusual weakness or fatigue, dizziness, headache, trouble breathing, dark urine, yellowing skin or eyes
- Liver injury—right upper belly pain, loss of appetite, nausea, light-colored stool, dark yellow or brown urine, yellowing skin or eyes, unusual weakness or fatigue
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Pros and cons of Pyrukynd (mitapivat)
Pros
Works well to treat symptoms of hemolytic anemia due to pyruvate kinase deficiency
Taken by mouth, with or without food
Stored at room temperature
Cons
No lower-cost generic available; brand name only
Interacts with a lot of commonly used medications
Not a good choice for people with liver problems
Pharmacist tips for Pyrukynd (mitapivat)
Take Pyrukynd (mitapivat) with or without food. If it's hurting your stomach, try taking it with food to help. Don't split, crush, chew, or dissolve the tablets.
If you miss a dose of Pyrukynd (mitapivat) by less than 4 hours, take the dose as soon as possible. If you missed your dose of Pyrukynd (mitapivat) by more than 4 hours, skip the missed dose. Start taking Pyrukynd (mitapivat) again at your next scheduled dose, then continue with your regular dosing schedule.
Don't suddenly stop taking Pyrukynd (mitapivat). Doing so can put you at risk for anemia. If you want to stop this medication, let your provider know. They can help you stop Pyrukynd (mitapivat) safely by slowly lowering your dose over time.
Pyrukynd (mitapivat) interacts with a lot of commonly used medications. Tell your provider about all of the medications you're taking, including over-the-counter and prescription medications as well as herbal supplements. Don't start taking any new medications without talking to your provider or pharmacist first.
Frequently asked questions about Pyrukynd (mitapivat)
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What are the risks and warnings for Pyrukynd (mitapivat)?
Pyrukynd (mitapivat) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Return of anemia symptoms
Don't suddenly stop taking Pyrukynd (mitapivat) without talking to your provider first. Doing so can cause short-term hemolytic anemia. If you'd like to stop taking Pyrukynd (mitapivat), talk to your provider to come up with a plan on how to lower your dose slowly over time until you completely stop taking it. Contact your provider if you experience any symptoms of hemolytic anemia, such as yellowing of skin or eyes, dark urine, dizziness, tiredness, trouble breathing.
Pyrukynd (mitapivat) dosage forms
| Dosage | Quantity | Price as low as | Price per unit |
|---|---|---|---|
| 28 day packs of 5mg | 1 carton | $25359.84 | $25359.84 |
| 28 day packs of 20mg | 1 carton | $25359.84 | $25359.84 |
| 28 day packs of 50mg | 1 carton | $25359.84 | $25359.84 |
| taper pack of 5mg | 1 carton | $3180.48 | $3180.48 |
| taper pack of 20mg/5mg | 1 carton | $6348.96 | $6348.96 |
| taper pack of 50mg/20mg | 1 carton | $6348.96 | $6348.96 |
Typical dosing for Pyrukynd (mitapivat)
The typical starting dose is 5 mg by mouth twice daily. Your provider will slowly raise your dose, usually every month, based on your hemoglobin levels. The maximum dose is 50 mg twice daily.
Your dose might be different if you have liver problems, if you're taking a medication that interacts with Pyrukynd (mitapivat), and if you're having side effects.
How much does Pyrukynd (mitapivat) cost?
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References
Best studies we foundAgios Pharmaceuticals, Inc. (2022). Pyrukynd- mitapivat tablet, film coated; mitapivat [package insert]. DailyMed.
Al-Samkari, H., et al. (2021). Mitapivat, a novel pyruvate kinase activator, for the treatment of hereditary hemolytic anemias. Therapeutics Advances in Hematology.
Al-Samkari, H., et al. (2022). Mitapivat versus placebo for pyruvate kinase deficiency. The New England Journal of Medicine.
American Red Cross. (n.d.). Red blood cells and why they are important.
Billet, H. H. (1990). Chapter 151: Hemoglobin and hematocrit. Clinical Methods: The History, Physical, and Laboratory Examinations. 3rd edition.
Israelsen, W. J., et al. (2015). Pyruvate kinase: Function, regulation and role in cancer. Seminars in Cell & Developmental Biology.
Memorial Sloan Kettering Cancer Center. (2021). Facts about blood and blood cells .
National Heart, Lung, and Blood Institute. (2022). Hemolytic anemia.
National Organization for Rare Disorders. (2023). Pyruvate kinase deficiency.
U.S. Food and Drug Administration. (2023). Drug trials snapshot: Pyrukynd.
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